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Written question asked by Lord Garnier (Conservative) on Thursday, 5 February 2015, in the House of Commons. It was due for an answer on Monday, 9 February 2015. It was answered by George Freeman (Conservative) on Wednesday, 11 February 2015 on behalf of the Department of Health.

Muscular Dystrophy: Drugs

Question

To ask the Secretary of State for Health, what discussions he has had with the National Institue for Health and Care Excellence on NHS commissioning of the drug Translarna, in response to the conditional approval given by the European Commission to that drug; when he plans for his review of the pathways for the development, assessment, and adoption of innovative medicines and medical technology to be completed; and if he will make a statement.

Answer

Following discussions between the Department, NHS England and the National Institute for Health and Care Excellence (NICE), it was decided that Translarna (ataluren) for treating Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene should be considered for formal referral to NICE’s highly specialised technologies (HST) programme which evaluates treatments for very rare conditions. NICE has recently consulted stakeholders on the draft remit and scope of its proposed HST evaluation of Translarna.

Ministers will take a decision on the formal referral of this topic following the outcome of NICE’s scoping work.

The review of the pathway for the development, assessment, and adoption of innovative medicines and medical technologies will consider how to speed up access for National Health Service patients to cost-effective new diagnostics, medicines and devices. We anticipate an initial report before the end of the year.

Secondary information

Type
Written question
Reference
223636
Session
2014-15
Subjects
Drugs Muscular dystrophy Ataluren
Link
View this Written question on www.parliament.uk